Shares of Agios Pharmaceuticals (AGIO) soared 14.5% after the company reported positive safety and clinical data on pipeline candidate, AG-120, from an ongoing phase I dose escalation study.
AG-120 is being evaluated for treating patients with IDH1-mutant positive advanced hematologic malignancies, including acute myeloid leukemia (AML.V).
The ongoing trial enrolled 17 patients as of Oct 17, 2014. These patients showed documented IDH1 mutation whose cancer relapsed or failed to respond (refractory) to at least one prior treatment regimen.
Out of these 17 patients, only 14 patients with relapsed and/or refractory AML were evaluable as three patients had recently initiated therapy.
Initial data from the study showed that four patients achieved complete remissions along with early evidence of durability. Responses across the four dose levels showed that AG-120 was well tolerated, with the majority of adverse events reported between mild to moderate. Meanwhile, one additional patient remains stable.
Data revealed that AG-120 showed favorable drug exposure and pharmacokinetics at all doses tested. AG-120 was effective in substantially reducing plasma levels of the oncometabolite 2-hydroxyglutarate (2HG), which is produced by the mutant IDH1 protein, to the level observed in healthy volunteers.
Based on positive initial data, Agios Pharmaceuticals plans to initiate multiple expansion cohorts in the first half of 2015.
We note Agios is developing AG-120 under a collaboration agreement with Celgene Corp. (CELG) with primary focus on cancer metabolism. Agios is also developing AG-221 under this agreement with Celgene.
While Agios has development and commercial rights for AG-120 in the U.S., Celgene has an exclusive option to rights outside the U.S.
The positive initial data on AG-120 will be a great boost for the development plan of the candidate. We expect investor focus to remain on data from both these candidates. Moreover, we are positive about the company’s partnership with Celgene, a key player in the cancer market. We believe Celgene’s experience in the field will help Agios to drive the candidates.
Agios currently carries a Zacks Rank #3 (Hold). Other well-placed stocks in the health care sector include Biogen Idec (BIIB) and Impax Laboratories Inc. (IPXL). Both carry a Zacks Rank #1 (Strong Buy).